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Pemphigus VulgarisPhase 2NCT03334058

A Study to Evaluate the Safety, PD, PK and Efficacy of ARGX-113 in Patients With Pemphigus

A Phase 2 study testing ARGX-113 in people with Pemphigus Vulgaris and Pemphigus Foliaceus, run by argenx at 12 sites in 5 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The proposed study is an open-label, non-controlled, adaptive-design Phase II study to evaluate the safety, pharmacodynamics, pharmacokinetics, efficacy, and conditions of use (dosage, frequency of administration at maintenance) of ARGX-113 in patients with mild to moderate Pemphigus (Vulgaris or Foliaceus), either… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Pemphigus Vulgaris, Pemphigus Foliaceus
What is being tested?
ARGX-113
Could I receive a placebo?
No, everyone receives an active treatment
How many people
34 participants
Where
12 sitesin 5 countries
Study dates
18 Oct 2017to 28 Oct 2020
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Male or female patients aged ≥ 18 years.
  • Clinical diagnosis of PV or PF, that has been confirmed by positive direct immunofluorescence and by positive indirect immunofluorescence and/or ELISA.
  • Mild to moderate disease severity (PDAI < 45).
  • Newly diagnosed patients or relapsing patients off therapy with or without a first course of prednisone of maximum 4 weeks, and for whom an initial period of ARGX-113 monotherapy is judged clinically acceptable; or newly diagnosed patients or relapsing patients off therapy on a first course of oral prednisone at stable dose for at least 2 weeks and for whom ARGX-113 monotherapy is considered not clinically acceptable; or patients who relapse despite oral prednisone at tapered dose +/- a conventional immunosuppressant (e.g. azathioprine, mycophenolate mofetil).
  • Identified serum levels of autoantibodies directed against Dsg 3 and/or Dsg-1 antigens at screening, using indirect immunofluorescence or ELISA.
  • Ability to understand the requirements of the study, provide written informed consent (including consent for the use and disclosure of research-related health information), and comply with the study protocol procedures (including required study visits).

Who cannot join

  • Pregnant and lactating women, and those intending to become pregnant during the study or within 90 days after the last dosing. Women of childbearing potential should have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline, prior to administration of IMP.
  • Male patients who are sexually active that do not intend to use effective methods of contraception during the study or within 90 days after the last dosing.
  • Confirmed diagnosis of paraneoplastic pemphigus, drug-induced pemphigus or any other non-PV/non-PF autoimmune blistering disease.
  • History of refractory disease to active third line therapy (e.g. intravenous polyvalent human immunoglobulins [IVIg], rituximab, plasma exchange/ immunoadsorption).
  • Use of therapies other than oral prednisone and conventional immunosuppressants, that can interfere in the clinical course of the disease (e.g. intravenous [IV] prednisolone bolus, dapsone, sulfasalazine, tetracyclines, nicotinamide, plasmapheresis/ plasma exchange, immunoadsorption and IVIg) within 2 months prior to Baseline visit.
  • Use of rituximab and other CD20 target biologics within 6 months prior to Baseline visit.
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  • History of anaphylactic reaction, or a known hypersensitivity reaction to one of the components of the IMP.
  • History of vaccination within the last 4 weeks prior to Baseline visit, or with a planned vaccination during the study, with the exception of seasonal vaccination (e.g. influenza vaccine).
  • Recent serious infection (i.e., requiring injectable antimicrobial therapy or hospitalization) within the 8 weeks prior to Baseline visit.
  • Known active or chronic viral infection with hepatitis B virus (HBV); refer to the Centers for Disease Control and Prevention (CDC) guidelines.
  • Known seropositive or active infection with hepatitis C virus (HCV).
  • Known history of or known viral infection with human immunodeficiency virus (HIV 1 and 2 antibodies).
  • Body Mass Index (BMI) at Screening > 35,0 kg/m2.
  • Clinical evidence of significant active, unstable or uncontrolled concomitant disease (e.g. cardiovascular, pulmonary, hematologic, gastrointestinal, endocrinological and metabolic, hepatic, renal, neurologic, malignancy, infectious diseases, coagulopathies, other autoimmune disease) or condition (lack of peripheral venous access, recent major surgery, etc), which, in the opinion of the investigator, puts the patient at undue risk or may affect the interpretation of the results.
  • Patients in general health condition not allowing study participation (Karnofsky index < 60%; see Appendix 14.2).
  • At Screening, have clinically significant laboratory abnormalities as below:
  • Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3 × upper limit of normal (ULN)
  • Total serum bilirubin of > 1.5 x ULN (except for Grade 1 hyperbilirubinemia as defined by the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE), due solely to a documented medical diagnosis of Gilbert's syndrome)
  • Serum creatinine of > 1.5 mg/dL or creatinine clearance < 50 mL/min (using the Chronic Kidney Disease Epidemiology Creatinine formula)
  • Hemoglobin (Hb) ≤ 9 g/dL
  • International normalized ratio (INR) > 1.5 or activated partial thromboplastin time (aPTT) > 1.5 x ULN
  • Total immunoglobulin G (IgG) level < 6 g/L
  • Presence of > 1 + proteinuria dipstick
  • Patient having participated in another interventional study within the last 3 months prior to Baseline visit.

Trial locations and contacts

12 sites in 5 countries.

12 sites in 5 countries

Hungary3 sites

  • University of Debrecen Medical Center Department of Dermatology

    Debrecen

  • University of Pécs Clinical Center , Department of Dermatology, Venerology and Oncodermatology

    Pécs

  • University of Szeged Faculty of Medicine Albert Szent-Györgyi Medical Center Department of Dermatology and Allergology

    Szeged

Other trials in Hungary

Israel3 sites

  • HaEmek Medical center, Dermatology Department

    Afula

  • Department of Dermatology, The Chaim Sheba Medical Center

    Tel Aviv

  • Department of dermatology, The Tel Aviv Sourasky Medical Center

    Tel Aviv

Other trials in Israel

Germany2 sites

  • University of Lübeck and UKSH, Department of Dermatology and Lübeck Institute of Experimental Dermatology

    Lübeck

  • Clinic of Dermatology and Allergology - Philipps University Marburg

    Marburg

Other trials in Germany

Italy2 sites

  • Dermopathic Institute of the Immaculate - Foundation "Luigi Maria Monti"

    Rome

  • Foundation Policlinico A. Gemelli - Dermatology Department

    Rome

Other trials in Italy

Ukraine2 sites

  • National Medical University named after O.O.Bohomolets, Department of Dermatology and Venereology based on Oleksandrivska Clinical Hospital of Kyiv City, Department of Dermatology

    Kyiv

  • Municipal Institution "Zaporizhzhya Regional Dermatology and Venereology Clinical Dispensary" of Zaporizhzhya Regional Council

    Zaporizhzhya

Other trials in Ukraine

About this study

Official title: An Open-label, Non-controlled, Phase II Study to Evaluate the Safety, Pharmacodynamics, Pharmacokinetics, Efficacy and Conditions of Use of ARGX-113 in Patients With Mild to Moderate Pemphigus (Vulgaris and Foliaceus)

The proposed study is an open-label, non-controlled, adaptive-design Phase II study to evaluate the safety, pharmacodynamics, pharmacokinetics, efficacy, and conditions of use (dosage, frequency of administration at maintenance) of ARGX-113 in patients with mild to moderate Pemphigus (Vulgaris or Foliaceus), either newly diagnosed or relapsing.

The total study duration for each patient is less than 6 months. It consists of a Screening period, an Induction, a maintenance treatment period followed by a treatment-free Follow-up (FU) period.

Being tested
ARGX-113
Medical subject headings
Pemphigus

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ARGX-113Drug

    human IgG1-derived Fc fragment that binds to human neonatal Fc receptor (FcRn)

Study groups in NCT03334058
Study groupRoleWhat they receive
ARGX-113ExperimentalNot described
Receives: Drug: ARGX-113

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Safety and tolerability as measured by the incidence and severity of treatment-emergent (serious) adverse events over the study.

    Measured: Up to 6 months

Secondary outcomes (8)

  1. Evaluation of serum levels of total IgG and subtypes (IgG1, IgG2, IgG3, IgG4)

    Measured: Up to 6 months

  2. Evaluation of serum levels of anti Dsg-1 and -3 autoantibodies

    Measured: Up to 6 months

  3. Pemphigus Disease Area Index (PDAI)

    The score has a range from 0 to 263, the higher the score, the more severe the disease.

    Measured: Up to 6 months

  4. Time to disease control (DC), control being defined as the absence of new lesions and established lesions beginning to heal

    Measured: Up to 6 months

  5. Time until relapse, relapse being defined as the appearance of 3 or more new lesions a month that do not heal spontaneously within 1 week, or as the extension of established lesions

    Measured: Up to 6 months

  6. Pharmacokinetic parameters of ARGX 113: Tmax

    Measured: Up to 6 months

  7. Pharmacokinetic parameters of ARGX 113: Cmax

    Measured: Up to 6 months

  8. Incidence of anti-drug antibodies (ADA) to ARGX 113

    Measured: Up to 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted7 Nov 2017
  2. Study start18 Oct 2017
  3. Primary completion28 Oct 2020
  4. Study completion28 Oct 2020

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
34 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03334058
Sponsor's protocol ID
ARGX-113-1701
Other IDs
2017-002333-40, 2017
Status last verified
Nov 2020
First submitted
23 Oct 2017
Last updated
14 Dec 2020
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No

Publications

  • Maho-Vaillant M, Sips M, Golinski ML, Vidarsson G, Goebeler M, Stoevesandt J, Bata-Csorgo Z, Balbino B, Verheesen P, Joly P, Hertl M, Calbo S. FcRn Antagonism Leads to a Decrease of Desmoglein-Specific B Cells: Secondary Analysis of a Phase 2 Study of Efgartigimod in Pemphigus Vulgaris and Pemphigus Foliaceus. Front Immunol. 2022 May 18;13:863095. doi: 10.3389/fimmu.2022.863095. eCollection 2022.

    PubMed 35663943

  • Goebeler M, Bata-Csorgo Z, De Simone C, Didona B, Remenyik E, Reznichenko N, Stoevesandt J, Ward ES, Parys W, de Haard H, Dupuy P, Verheesen P, Schmidt E, Joly P; ARGX-113-1701 Investigator Study Group. Treatment of pemphigus vulgaris and foliaceus with efgartigimod, a neonatal Fc receptor inhibitor: a phase II multicentre, open-label feasibility trial. Br J Dermatol. 2022 Mar;186(3):429-439. doi: 10.1111/bjd.20782. Epub 2021 Nov 28.

    PubMed 34608631

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ARGX-113 in people with Pemphigus Vulgaris and Pemphigus Foliaceus, run by argenx at 12 sites in 5 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 6 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 14 Dec 2020. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 12 sites in Hungary, Israel, Germany, Italy, Ukraine. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Safety and tolerability as measured by the incidence and severity of treatment-emergent (serious) adverse events over the study. (time frame: Up to 6 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03334058, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Dec 2020.

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